Abstract: The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.

Abstract: Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.

Abstract: Provided herein are RNAi molecules for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

Abstract: Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.

Abstract: The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.

Abstract: The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.

Abstract: Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.

Abstract: The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.

Abstract: The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.

Abstract: The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.

Abstract: The invention is related to methods of producing rod-derived cone viability factor (RdCVF). This invention also relates to the treatment of an ocular disease in a mammal using RdCVF. Also provided are expression vectors for high secreted expression of RdCVF of using nucleotide sequences encoding heterologous signal proteins and optionally markers for furin cleavage.

Abstract: The present invention relates to polymer-modified viruses, processes for obtaining them and their use. The invention also provides means of attaching polymer molecules to viral particles whereas retaining infectivity of the modified virus.

Abstract: Viruses are modified by coupling a polymer such as polyethylene glycol to obtain polymer-modified viruses that can exhibit reduced antigenicity while retaining infectivity, and which may exhibit increased circulation time in vivo. The polymer may be directly covalently attached or indirectly covalently attached via an intermediate coupling moiety to the virus. The polymer may also be indirectly noncovalently attached to the virus via a ligand such as an antibody having specificity for a viral surface component. To prepare the polymer-modified virus, the polymer is activated and coupled to the virus. A preferred activated polymer is tresyl-monomethoxypolyethylene glycol having an average molecular weight of about 5000 daltons. The polymer-modified viruses have utility for therapeutic and diagnostic in vivo applications, and may be used to introduce a transgene into a target cell by infection, or be administered to a subject having a tumor where the polymer-modified virus localizes to the tumor.

Abstract: The present invention relates to polymer-modified viruses, processes for obtaining them and their use. The invention also provides means of attaching polymer molecules to viral particles whereas retaining infectivity of the modified virus.

Abstract: This invention describes a nucleic acid delivery vehicle construct for transfecting and/or infecting a target cell. The construct is made of a delivery vehicle and a bifunctional complex for linking the delivery vehicle to a target cell. The bifunctional complex has a delivery vehicle-binding molecule or fragment (“delivery vehicle-binding portion”), a molecule or fragment thereof that binds to a cell surface molecule on the target cell (“cell surface molecule-binding portion”) and a linker which connects the molecules or fragments.

Abstract: The present invention provides a method for purifying a membrane associated protein, e.g., cystic fibrosis transmembrane conductance regulator (hereinafter CFTR) in a functional form. The method involves contacting a membrane-associated protein-membrane fraction complex with a detergent forming a solubilized complex and chromatographically isolating the membrane-associated protein from the solubilized complex in a functional form. The functional form of the purified membrane-associated protein of the present invention preferably is sufficiently pure to allow its introduction into humans for therapeutic purposes.